Pietro Refolo

Testing the HTA Core Model: Experiences from two pilot projects

OBJECTIVES The aim of this study was to analyze and describe process and outcomes of two pilot assessments based on the HTA Core Model, discuss the applicability of the model, and explore areas of development. METHODS Data were gathered from HTA Core Model and pilot Core HTA documents, their validation feedback, questionnaires to investigators, meeting minutes, emails, and discussions in the coordinating team meetings in the Finnish Office for Health Technology Assessment (FINOHTA). RESULTS The elementary structure of the HTA Core Model proved useful in preparing HTAs. Clear scoping and good coordination in timing and distribution of work would probably help improve applicability and avoid duplication of work. CONCLUSIONS The HTA Core Model can be developed into a platform that enables and encourages true HTA collaboration in terms of distribution of work and maximum utilization of a common pool of structured HTA information for national HTA reports.

Health technology assessment (HTA): ethical aspects

Abstract“HTA is a multidisciplinary process that summarizes information about the medical, social, economic and ethical issues related to the use of a health technology in a systematic, transparent, unbiased, robust manner. Its aim is to inform the formulation of safe, effective, health policies that are patient focused, and seek to achieve best value” (EUnetHTA 2007). Even though the assessment of ethical aspects of a health technology is listed as one of the objectives of a HTA process, in practice, the integration of these dimensions into reports remains limited. The article is focused on four points: 1. the HTA concept; 2. the difficult HTA-ethics relationship; 3. the ethical issues in HTA; 4. the methods for integrating ethical analysis into HTA.

INTEGRATING ETHICS IN HEALTH TECHNOLOGY ASSESSMENT: MANY WAYS TO ROME

OBJECTIVES The aim of this study was to identify and discuss appropriate approaches to integrate ethical inquiry in health technology assessment (HTA). METHODS The key question is how ethics can be integrated in HTA. This is addressed in two steps: by investigating what it means to integrate ethics in HTA, and by assessing how suitable the various methods in ethics are to be integrated in HTA according to these meanings of integration. RESULTS In the first step, we found that integrating ethics can mean that ethics is (a) subsumed under or (b) combined with other parts of the HTA process; that it can be (c) coordinated with other parts; or that (d) ethics actively interacts and changes other parts of the HTA process. For the second step, we found that the various methods in ethics have different merits with respect to the four conceptions of integration in HTA. CONCLUSIONS Traditional approaches in moral philosophy tend to be most suited to be subsumed or combined, while processual approaches being close to the HTA or implementation process appear to be most suited to coordinated and interactive types of integration. The article provides a guide for choosing the ethics approach that appears most appropriate for the goals and process of a particular HTA.

Lay and professional stakeholder involvement in scoping palliative care issues: Methods used in seven European countries

Background: Stakeholders are people with an interest in a topic. Internationally, stakeholder involvement in palliative care research and health technology assessment requires development. Stakeholder involvement adds value throughout research (from prioritising topics to disseminating findings). Philosophies and understandings about the best ways to involve stakeholders in research differ internationally. Stakeholder involvement took place in seven countries (England, Germany, Italy, Lithuania, the Netherlands, Norway and Poland). Findings informed a project that developed concepts and methods for health technology assessment and applied these to evaluate models of palliative care service delivery. Aims: To report on stakeholder involvement in the INTEGRATE-HTA project and how issues identified informed project development. Design: Using stakeholder consultation or a qualitative research design, as appropriate locally, stakeholders in seven countries acted as ‘advisors’ to aid researchers’ decision making. Thematic analysis was used to identify key issues across countries. Setting/participants: A total of 132 stakeholders (82 professionals and 50 ‘lay’ people) aged ⩾18 participated in individual face-to-face or telephone interviews, consultation meetings or focus groups. Results: Different stakeholder involvement methods were used successfully to identify key issues in palliative care. A total of 23 issues common to three or more countries informed decisions about the intervention and comparator of interest, sub questions and specific assessments within the health technology assessment. Conclusion: Stakeholders, including patients and families undergoing palliative care, can inform project decision making using various involvement methods according to the local context. Researchers should consider local understandings about stakeholder involvement as views of appropriate and feasible methods vary. Methods for stakeholder involvement, especially consultation, need further development.

Health Technology Assessment of pathogen reduction technologies applied to plasma for clinical use.

Although existing clinical evidence shows that the transfusion of blood components is becoming increasingly safe, the risk of transmission of known and unknown pathogens, new pathogens or re-emerging pathogens still persists. Pathogen reduction technologies may offer a new approach to increase blood safety. The study is the output of collaboration between the Italian National Blood Centre and the Post-Graduate School of Health Economics and Management, Catholic University of the Sacred Heart, Rome, Italy. A large, multidisciplinary team was created and divided into six groups, each of which addressed one or more HTA domains.Plasma treated with amotosalen + UV light, riboflavin + UV light, methylene blue or a solvent/detergent process was compared to fresh-frozen plasma with regards to current use, technical features, effectiveness, safety, economic and organisational impact, and ethical, social and legal implications. The available evidence is not sufficient to state which of the techniques compared is superior in terms of efficacy, safety and cost-effectiveness. Evidence on efficacy is only available for the solvent/detergent method, which proved to be non-inferior to untreated fresh-frozen plasma in the treatment of a wide range of congenital and acquired bleeding disorders. With regards to safety, the solvent/detergent technique apparently has the most favourable risk-benefit profile. Further research is needed to provide a comprehensive overview of the cost-effectiveness profile of the different pathogen-reduction techniques. The wide heterogeneity of results and the lack of comparative evidence are reasons why more comparative studies need to be performed.

Ethical analysis in HTA of complex health interventions

BackgroundIn the field of health technology assessment (HTA), there are several approaches that can be used for ethical analysis. However, there is a scarcity of literature that critically evaluates and compares the strength and weaknesses of these approaches when they are applied in practice. In this paper, we analyse the applicability of some selected approaches for addressing ethical issues in HTA in the field of complex health interventions. Complex health interventions have been the focus of methodological attention in HTA. However, the potential methodological challenges for ethical analysis are as yet unknown.MethodsSix of the most frequently described and applied ethical approaches in HTA were critically assessed against a set of five characteristics of complex health interventions: multiple and changing perspectives, indeterminate phenomena, uncertain causality, unpredictable outcomes, and ethical complexity. The assessments are based on literature and the authors’ experiences of developing, applying and assessing the approaches.ResultsThe Interactive, participatory HTA approach is by its nature and flexibility, applicable across most complexity characteristics. Wide Reflective Equilibrium is also flexible and its openness to different perspectives makes it better suited for complex health interventions than more rigid conventional approaches, such as Principlism and Casuistry. Approaches developed for HTA purposes are fairly applicable for complex health interventions, which one could expect because they include various ethical perspectives, such as the HTA Core Model® and the Socratic approach.ConclusionThis study shows how the applicability for addressing ethical issues in HTA of complex health interventions differs between the selected ethical approaches. Knowledge about these differences may be helpful when choosing and applying an approach for ethical analyses in HTA. We believe that the study contributes to increasing awareness and interest of the ethical aspects of complex health interventions in general.

Ethical and deontological issues in Transfusion Medicine

Recent developments in Transfusion Medicine Since the second half of the 1980s, Transfusion Medicine has gradually taken on an increasing autonomous place in the world of medical specialties, continuing its evolution from its initial, mainly immunohaematological role, towards new diagnostic, clinical-therapeutic and research activities. Currently, in addition to direct care activities for patients and support of specific highly specialised care processes, Transfusion Medicine ranges from the collection and banking of haematopoietic stem cells to the banking of tissues and cell therapy. In parallel to the growth of the activities mentioned above, there has been constant progress in research activities, as evidenced by the exponential increase in randomised controlled trials and meta-analyses1. Despite this gradual transformation, blood collection and the production of sufficient amounts of blood components to reach and/or maintain local and national self-sufficiency continue to be priority aims and the object of planning the activities of the whole national transfusion network2. Blood donation and Transfusion Medicine now constitute an essential component of the integrated care processes that are typical of the current, modern vision of health, oriented in a logic of disease management. Operational coordination and management, including administrative area, of the many activities that are part of the transfusion process have a fundamental role in the clinical governance of all the procedures pertaining to the field of transfusion3. Blood and blood components are in fact “the final result of various processes (clinical, laboratory, social), which are linked together and intertwine, involving different professional skills and responsibilities”4. The transfusion process can be defined as a “supply chain” because, ultimately, the patients’ demands condition the supply, that is, the need for blood donations. A lack of blood occurs when the supply is not sufficient to meet the demand and the transfusion needs of the patients cannot be met quantitatively and/or qualitatively, or waiting times are imposed, particularly for elective surgery. The supply chain in the transfusion field begins with the donor and the donor procedures and continues through the processing, qualification and biological validation, reception of transfusion requests, evaluation of the appropriateness of the requests, determination of the immunological compatibility, allocation of blood components to individual patients, delivery to the requesting clinical unit, transfusion to the recipient patient and recording and reporting of any side effects and adverse events possibly associated with the transfusion5. The correct management of the processes that make up the transfusion supply chain affects the safety of the whole process, the monitoring of which is a specific aim of haemovigilance systems. A good haemovigilance system includes appropriate management of patients’ transfusion requirements as well as safe fulfilment of these needs. Transfusion safety goes beyond the intrinsic safety of the transfused therapeutic products, depending on a series of closely interconnected processes which start with the donor and finish with the recipient. The systematic recording of transfusion outcomes and adverse events associated with transfusion therapy constitutes the afferent branch of every haemovigilance system and plays a fundamental role in the search for and the identification of possible strategies for improvement. In contrast, the purpose of the efferent branch of these systems is to achieve a constant, global improvement in the transfusion process through an analysis of the data collected and their possible changes over time, to identify possible corrective or preventive strategies based on objective evidence. Therefore, it seems clear that the multifaceted and complex transfusion process, with its unique global organisational and management framework, is not possible without a systematic approach aimed at raising quality and, therefore, at continuous improvement4. Since 2002, and in particular in the 3-year period from 2005 to 2007, national and European community law-making in the field of blood transfusion, and related activities, has been more intense and important than ever before6–18. The regulatory scenario that has developed is, therefore, very complex in terms of relations between European and national legislations, in particular because of the need to harmonise the acts implementing the European Directives with existing national laws. The European regulatory provisions concerning transfusions and associated activities are strongly inspired by the social and ethical concepts of solidarity, voluntariness and periodic repetition of donations, as well as the fundamental principle of protecting public health, in terms of fairness, transparency and right of access to safe and systematically controlled services19. Great emphasis is also given to the principles of management and control of the specific care processes, in order that these are aimed at ensuring high organisational and professional standards as well as safeguarding the appropriateness of the clinical use of blood products. Finally, specific regulations concerning the cord blood banking have been introduced20. The aim of this review is to focus on the ethical and deontological issues of the macro-processes of the transfusion chain, extending the considerations to the most recent, innovate area of development of Transfusion Medicine, biobanking, and outlining possible additional areas of practical ethical reflection related to it, such as health technology assessment and the organisational ethics of services. The reason for this is the paucity of specific contributions on the subject. The ethical issues related to the donation of cord blood will not be dealt with here: the reader is referred to the recent, but still limited literature21–22.

Taking patient heterogeneity and preferences into account in health technology assessments

OBJECTIVES The INTEGRATE-HTA project provided methodology to evaluate complex technologies. This study provides guidance on how to retrieve and critically appraise available evidence on moderators and predictors of treatment effects and on patient preferences for treatment outcomes as a source of complexity. METHODS Search filters for PubMed were developed by hand-searching a large volume of articles reporting on relevant aspects. Search terms were retrieved from selected papers and algorithmically combined to find the optimal combination of search terms. For the development of the appraisal checklists literature was searched in PubMed and Google Scholar together with citation chasing. For the CHecklist for the Appraisal of Moderators and Predictors (CHAMP) a Delphi procedure was used to value a set of eligible appraisal criteria retrieved from the literature. RESULTS Search filters were developed optimized for different accuracy measures. The final version of CHAMP consists of a seventeen questions covering the design, analysis, results and transferability of results of moderator and predictor analysis. The final checklist for appraisal of literature on patient preferences for treatment outcomes consist of six questions meant to help the user to identify relevant quality issues together with a guidance toward existing tools concerning the appraisal of specific preference elicitation methods. CONCLUSIONS Incorporating knowledge on subgroups for whom a specific treatment will produce more benefit holds the promise of better targeting and, ultimately, enhancing overall effectiveness and efficiency of healthcare technology. Finally, incorporating information on preferences for treatment outcomes will foster health technology assessment that addresses outcomes that are important to patients.

Structured methodology review identified seven (RETREAT) criteria for selecting qualitative evidence synthesis approaches

OBJECTIVE To compare and contrast different methods of qualitative evidence synthesis (QES) against criteria identified from the literature and to map their attributes to inform selection of the most appropriate QES method to answer research questions addressed by qualitative research. STUDY DESIGN AND SETTING Electronic databases, citation searching, and a study register were used to identify studies reporting QES methods. Attributes compiled from 26 methodological papers (2001-2014) were used as a framework for data extraction. Data were extracted into summary tables by one reviewer and then considered within the author team. RESULTS We identified seven considerations determining choice of methods from the methodological literature, encapsulated within the mnemonic Review question-Epistemology-Time/Timescale-Resources-Expertise-Audience and purpose-Type of data. We mapped 15 different published QES methods against these seven criteria. The final framework focuses on stand-alone QES methods but may also hold potential when integrating quantitative and qualitative data. CONCLUSION These findings offer a contemporary perspective as a conceptual basis for future empirical investigation of the advantages and disadvantages of different methods of QES. It is hoped that this will inform appropriate selection of QES approaches.

Electronic Medical Diary (EMD): Ethical Analysis in a HTA Process

Ethical analysis within Health Technology Assessment (HTA) – a comprehensive form of health policy research that examines the short- and long- term consequences of the application or use of technologies (in a broad meaning) – aims at analysing the moral questions raised by the technology itself and by the consequences of implementing or not a health technology as well as ethical issues that are inherent in the HTA process. The work intends to assess, within a HTA process, the ethical consequences of implementing the Electronic Medical Diary (EMD) in health care systems. The EMD is a device for supporting the daily registration and collection of clinical events related to a certain patient. The storage of these patient-specific clinical data constitutes the patient database (PDB) that may be connected with the many online tools which can improve the flow of information within the hospital information system. Such devices should be able to replace the traditional paper record.